TWI787163B - 用於減少tau表現之組合物及方法 - Google Patents

用於減少tau表現之組合物及方法 Download PDF

Info

Publication number
TWI787163B
TWI787163B TW105142107A TW105142107A TWI787163B TW I787163 B TWI787163 B TW I787163B TW 105142107 A TW105142107 A TW 105142107A TW 105142107 A TW105142107 A TW 105142107A TW I787163 B TWI787163 B TW I787163B
Authority
TW
Taiwan
Prior art keywords
dna
note
source
description
artificial sequence
Prior art date
Application number
TW105142107A
Other languages
English (en)
Chinese (zh)
Other versions
TW201733597A (zh
Inventor
歐芬吉姆 曼努埃拉 波里多羅
珍 韋勒
Original Assignee
瑞士商諾華公司
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 瑞士商諾華公司 filed Critical 瑞士商諾華公司
Publication of TW201733597A publication Critical patent/TW201733597A/zh
Application granted granted Critical
Publication of TWI787163B publication Critical patent/TWI787163B/zh

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Genetics & Genomics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Psychology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
TW105142107A 2015-12-21 2016-12-19 用於減少tau表現之組合物及方法 TWI787163B (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562270165P 2015-12-21 2015-12-21
US62/270,165 2015-12-21

Publications (2)

Publication Number Publication Date
TW201733597A TW201733597A (zh) 2017-10-01
TWI787163B true TWI787163B (zh) 2022-12-21

Family

ID=57758674

Family Applications (1)

Application Number Title Priority Date Filing Date
TW105142107A TWI787163B (zh) 2015-12-21 2016-12-19 用於減少tau表現之組合物及方法

Country Status (32)

Country Link
US (2) US11473083B2 (ru)
EP (1) EP3394259B1 (ru)
JP (3) JP7027328B2 (ru)
KR (1) KR20180089517A (ru)
CN (2) CN109072237B (ru)
AR (1) AR107579A1 (ru)
AU (2) AU2016376034B2 (ru)
BR (1) BR112018012304A2 (ru)
CA (1) CA3008094A1 (ru)
CL (3) CL2018001652A1 (ru)
CO (1) CO2018006297A2 (ru)
CR (1) CR20200609A (ru)
CU (1) CU24574B1 (ru)
DK (1) DK3394259T3 (ru)
DO (1) DOP2018000151A (ru)
EA (2) EA036417B1 (ru)
EC (1) ECSP18050575A (ru)
FI (1) FI3394259T3 (ru)
HK (1) HK1254822A1 (ru)
IL (3) IL305866A (ru)
JO (2) JOP20200228A1 (ru)
MX (2) MX2018007726A (ru)
MY (1) MY191737A (ru)
PE (1) PE20181448A1 (ru)
PH (1) PH12018501207A1 (ru)
RU (1) RU2747734C2 (ru)
SA (1) SA518391831B1 (ru)
SG (2) SG10201913434PA (ru)
SV (1) SV2018005714A (ru)
TW (1) TWI787163B (ru)
UY (1) UY37038A (ru)
WO (1) WO2017109679A1 (ru)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2013203395A1 (en) 2012-03-30 2013-10-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome
TW202246503A (zh) 2013-07-19 2022-12-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
JOP20190065A1 (ar) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau
CN111868068A (zh) * 2018-03-13 2020-10-30 詹森药业有限公司 用于在tau蛋白病变治疗中使用的经修饰的寡核苷酸
WO2020007892A1 (en) 2018-07-03 2020-01-09 F. Hoffmann-La Roche Ag Oligonucleotides for modulating tau expression
US20230203486A1 (en) * 2020-03-30 2023-06-29 Alnylam Pharmaceuticals, Inc. MICROTUBULE ASSOCIATED PROTEIN TAU (MAPT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
CN113817728A (zh) * 2020-06-19 2021-12-21 四川大学华西医院 一种有效干扰Tau蛋白表达的重组慢病毒及其应用
EP4355878A1 (en) * 2021-06-16 2024-04-24 Empirico Inc. Treatment of mtres1 related diseases and disorders
WO2024091874A1 (en) * 2022-10-25 2024-05-02 Empirico Inc. Gene therapy treatment of mtres1 related diseases and disorders

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW201503892A (zh) * 2013-07-19 2015-02-01 Isis Pharmaceuticals Inc 用於調節τ蛋白表現之組合物

Family Cites Families (93)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL154598B (nl) 1970-11-10 1977-09-15 Organon Nv Werkwijze voor het aantonen en bepalen van laagmoleculire verbindingen en van eiwitten die deze verbindingen specifiek kunnen binden, alsmede testverpakking.
US3817837A (en) 1971-05-14 1974-06-18 Syva Corp Enzyme amplification assay
US3939350A (en) 1974-04-29 1976-02-17 Board Of Trustees Of The Leland Stanford Junior University Fluorescent immunoassay employing total reflection for activation
US3996345A (en) 1974-08-12 1976-12-07 Syva Company Fluorescence quenching with immunological pairs in immunoassays
US4275149A (en) 1978-11-24 1981-06-23 Syva Company Macromolecular environment control in specific receptor assays
US4277437A (en) 1978-04-05 1981-07-07 Syva Company Kit for carrying out chemically induced fluorescence immunoassay
US4366241A (en) 1980-08-07 1982-12-28 Syva Company Concentrating zone method in heterogeneous immunoassays
BR9105935A (pt) 1990-01-11 1992-11-17 Isis Pharmaceuticals Inc Composicao,analogo de oligonucleotideo,oligonucleotideo sequenciado misto,processo para a preparacao de nucleosideos 2'-substituidos,composicao para modular a atividade de rna,processos para modular a producao de uma proteina por um organismo,para tratar um animal,para detectar a presenca ou ausencia de rna,oligonucleotideo ou analogo de analogo de oligonucleotideo resistente a nuclease e processo para tratar um organismo tendo uma doenca
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
US20020052331A1 (en) 1990-08-14 2002-05-02 Brenda Baker Compositions and methods for antisense inhibition of protein translation
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
ES2136654T3 (es) 1991-12-06 1999-12-01 Max Planck Gesellschaft Herramientas para el diagnostico y tratamiento de la enfermedad de alzheimer.
US7408027B1 (en) 1991-12-06 2008-08-05 Max-Planck-Gesellschaft Zur Forderung Der Wissenchaften Tools for the diagnosis and treatment of Alzheimer's disease
US5858784A (en) 1991-12-17 1999-01-12 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol- and liposome-based delivery
KR940021073A (ko) 1993-03-02 1994-10-17 미야베 요시가즈 알쯔하이머병의 예방 또는 치료제, 그 스크리닝 방법 및 인간 유래의 타우 단백질 키나아제 i
CA2116460A1 (en) 1993-03-02 1994-09-03 Mitsubishi Chemical Corporation Preventive or therapeutic agents for alzheimer's disease, a screening method of alzheimer's disease and tau-protein kinase i originated from human being
NZ266386A (en) 1993-05-11 1997-11-24 Univ North Carolina Use of antisense rna oligonucleotides in regulating gene expression
EP0882061B1 (en) 1996-02-14 2004-05-19 Isis Pharmaceuticals, Inc. Sugar-modified gapped oligonucleotides
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
DE04020014T1 (de) 1997-09-12 2006-01-26 Exiqon A/S Bi-zyklische - Nukleosid,Nnukleotid und Oligonukleotid-Analoga
US20040186071A1 (en) 1998-04-13 2004-09-23 Bennett C. Frank Antisense modulation of CD40 expression
US6475723B2 (en) 1998-05-01 2002-11-05 Mayo Foundation For Medical Education And Research Pathogenic tau mutations
US6664443B1 (en) 1998-05-01 2003-12-16 Mayo Foundation For Medical Education And Research Pathogenic Tau mutations in transgenic mice
KR20010083111A (ko) * 1998-07-14 2001-08-31 길리스 스티브 전립선암의 치료 및 진단을 위한 조성물 및 방법
US7098192B2 (en) * 1999-04-08 2006-08-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of STAT3 expression
US6350868B1 (en) 1999-04-26 2002-02-26 University Of North Carolina At Chapel Hill Antisense human fucosyltransferase sequences and methods of use thereof
CN101284873A (zh) * 1999-06-30 2008-10-15 科里克萨有限公司 用于肺癌治疗和诊断的组合物和方法
WO2001053340A2 (en) 2000-01-21 2001-07-26 Pharmacia & Upjohn Company Transgenic mouse model of human neurodegenerative disease
KR20030016233A (ko) * 2000-02-28 2003-02-26 디코드 제네틱스 이에이치에프 사람 정신분열증 유전자
WO2002055681A2 (en) 2001-01-11 2002-07-18 Bayer Ag Regulation of human tau-tubulin kinase
AU2003247521A1 (en) 2002-06-26 2004-01-19 Isis Pharmaceuticals, Inc. Antisense inhibition via rnase h-independent reduction in mrna
US20050130924A1 (en) 2002-06-26 2005-06-16 Monia Brett P. Antisense inhibition via RNAse H-independent reduction in mRNA
US20050106731A1 (en) 2002-08-05 2005-05-19 Davidson Beverly L. siRNA-mediated gene silencing with viral vectors
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
US20080176812A1 (en) 2002-08-05 2008-07-24 Davidson Beverly L Allele-specific silencing of disease genes
AU2003257850A1 (en) 2002-08-14 2004-03-03 Mitsubishi Kagaku Iatron, Inc. ANTIBODY SPECIFIC TO CENTRAL Tau-PROTEIN
US8163896B1 (en) 2002-11-14 2012-04-24 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
US7906326B2 (en) 2003-05-07 2011-03-15 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides associated with alzheimer's disease and uses thereof
US7217807B2 (en) 2002-11-26 2007-05-15 Rosetta Genomics Ltd Bioinformatically detectable group of novel HIV regulatory genes and uses thereof
US20070077553A1 (en) 2003-10-30 2007-04-05 Rosetta Genomics Bioinformatically detectable group of novel vaccinia regulatory genes and uses thereof
US20130130231A1 (en) 2002-11-26 2013-05-23 Isaac Bentwich Bioinformatically detectable group of novel viral regulatory genes and uses thereof
US20060257851A1 (en) 2002-11-26 2006-11-16 Itzhak Bentwich Bioinformatically detectable group of novel viral regulatory genes and uses thereof
US7696334B1 (en) 2002-12-05 2010-04-13 Rosetta Genomics, Ltd. Bioinformatically detectable human herpesvirus 5 regulatory gene
AU2003904328A0 (en) 2003-08-13 2003-08-28 Garvan Institute Of Medical Research Diagnosis and treatment of neurodegenerative disorders
US7888497B2 (en) 2003-08-13 2011-02-15 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof
MXPA06002216A (es) 2003-08-28 2006-04-27 Novartis Ag Interferencia de arn doble que tiene extremos romos y modificaciones 3'.
WO2005040382A2 (de) 2003-10-28 2005-05-06 Alexander Cherkasky Cherkasky - fusionsproteine enthaltend antikorperbinde-, antigenbinde - mikrotubulibinde und immunantwortauslosende
US20050244851A1 (en) 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
US7687616B1 (en) 2004-05-14 2010-03-30 Rosetta Genomics Ltd Small molecules modulating activity of micro RNA oligonucleotides and micro RNA targets and uses thereof
JP5697297B2 (ja) * 2004-05-14 2015-04-08 ロゼッタ ジノミクス リミテッド マイクロｎａｓおよびその使用
DK1766010T3 (da) 2004-06-28 2011-06-06 Univ Western Australia Antisense-oligonukleotider til induktion af exon-skipping og fremgangsmåder til anvendelse deraf
US7312035B2 (en) 2004-09-03 2007-12-25 Affymetrix, Inc. Methods of genetic analysis of yeast
CN101102789B (zh) 2004-12-03 2012-05-30 罗得岛医院 阿尔茨海默病的诊断和治疗
US7833513B2 (en) 2004-12-03 2010-11-16 Rhode Island Hospital Treatment of Alzheimer's Disease
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
US20070048756A1 (en) 2005-04-18 2007-03-01 Affymetrix, Inc. Methods for whole genome association studies
US8501703B2 (en) 2005-08-30 2013-08-06 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
US8258109B2 (en) 2005-10-20 2012-09-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of LMNA expression
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
US7901882B2 (en) 2006-03-31 2011-03-08 Affymetrix, Inc. Analysis of methylation using nucleic acid arrays
CA3042781C (en) 2006-04-03 2021-10-19 Roche Innovation Center Copenhagen A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
US20090280110A1 (en) 2006-06-27 2009-11-12 University Of Virginia Patent Foundation Cell Model for Alzheimer's Disease Pathology
CA2681568C (en) 2006-11-23 2019-01-08 Querdenker Aps Oligonucleotides for modulating target rna activity
EP2145014B1 (en) 2007-04-05 2012-12-12 The J. David Gladstone Institutes Agents that reduce neuronal overexcitation
JP2008307007A (ja) 2007-06-15 2008-12-25 Bayer Schering Pharma Ag 出生後のヒト組織由来未分化幹細胞から誘導したヒト多能性幹細胞
US8361980B2 (en) 2008-03-07 2013-01-29 Santaris Pharma A/S Pharmaceutical compositions for treatment of microRNA related diseases
EP2421970B1 (en) 2009-04-24 2016-09-07 Roche Innovation Center Copenhagen A/S Pharmaceutical compositions for treatment of hcv patients that are non-responders to interferon
KR102366851B1 (ko) 2009-11-12 2022-02-23 더 유니버시티 오브 웨스턴 오스트레일리아 안티센스 분자 및 이를 이용한 질환 치료방법
US20150018223A1 (en) 2010-01-28 2015-01-15 University Of Massachusetts Lowell Methods of diagnosing tau-associated neurodegenerative diseases
EP2550360B1 (en) 2010-03-24 2017-08-30 Mirrx Therapeutics A/s Bivalent antisense oligonucleotides
WO2012065143A1 (en) 2010-11-12 2012-05-18 The General Hospital Corporation Polycomb-associated non-coding rnas
US9145557B2 (en) 2010-11-23 2015-09-29 Mirrx Therapeutics A/S Oligonucleotides for modulation of target RNA activity
EP2655621B1 (en) 2010-12-20 2018-05-23 The General Hospital Corporation Polycomb-associated non-coding rnas
EP2702170B1 (en) * 2011-04-28 2015-12-16 Life Technologies Corporation Methods and compositions for multiplex pcr
WO2013013165A2 (en) 2011-07-21 2013-01-24 The Trustees Of The University Of Pennsylvania Use of lna compounds for therapeutic inhibition of mir-21 in systemic lupus erythematosus
AU2013203395A1 (en) * 2012-03-30 2013-10-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome
AU2013243953A1 (en) 2012-04-02 2014-10-30 Modernatx, Inc. Modified polynucleotides for the production of nuclear proteins
JP6321633B2 (ja) 2012-06-04 2018-05-09 ノバルティス アーゲー 部位特異的標識法およびそれによって生成される分子
US9695239B2 (en) 2012-10-24 2017-07-04 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Microtubule-modifying compound
US9200068B2 (en) 2012-12-18 2015-12-01 Regents Of The University Of Minnesota Compositions and methods related to tauopathy
JP2016502858A (ja) 2012-12-20 2016-02-01 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物
CA2906119A1 (en) 2013-03-14 2014-09-25 Isis Pharmaceuticals, Inc. Compositions and methods for modulating tau expression
LT2970964T (lt) 2013-03-14 2019-04-25 Sarepta Therapeutics, Inc. Kompozicijos su egzono praleidimu, skirtos raumenų distrofijos gydymui
WO2014153220A2 (en) 2013-03-14 2014-09-25 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
EP3007705A4 (en) 2013-06-12 2017-02-15 Oncoimmunin, Inc. Systemic in vivo delivery of oligonucleotides
WO2015068075A2 (en) 2013-10-24 2015-05-14 Institut National De La Sante Et De La Recherche Medicale Detection of tau
EP3169784B1 (en) * 2014-07-16 2020-06-10 Arrowhead Pharmaceuticals, Inc. Rnai compositions to treat apoc3-related diseases
US20170211064A1 (en) 2014-07-29 2017-07-27 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating tau expression
WO2016127002A1 (en) 2015-02-04 2016-08-11 Bristol-Myers Squibb Company Lna oligonucleotides with alternating flanks
EP3954993A1 (en) 2015-02-04 2022-02-16 Bristol-Myers Squibb Company Methods of selecting therapeutic molecules
EP3253875B1 (en) * 2015-02-04 2020-01-08 H. Hoffnabb-La Roche Ag Tau antisense oligomers and uses thereof
US20170035860A1 (en) * 2015-04-02 2017-02-09 Alexander C. Flynn Compositions and methods for treatment of neurogenerative diseases

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW201503892A (zh) * 2013-07-19 2015-02-01 Isis Pharmaceuticals Inc 用於調節τ蛋白表現之組合物

Also Published As

Publication number Publication date
EP3394259A1 (en) 2018-10-31
RU2018126545A3 (ru) 2020-10-12
EA036417B1 (ru) 2020-11-09
IL305866A (en) 2023-11-01
TW202311530A (zh) 2023-03-16
US20170175116A1 (en) 2017-06-22
UY37038A (es) 2017-07-31
EP3394259B1 (en) 2024-02-28
AR107579A1 (es) 2018-05-16
CA3008094A1 (en) 2017-06-29
SG11201804704PA (en) 2018-07-30
CL2019000149A1 (es) 2019-06-07
MX2023008211A (es) 2023-07-18
SA518391831B1 (ar) 2022-08-16
EA201891468A1 (ru) 2018-12-28
DK3394259T3 (da) 2024-05-27
CN109072237B (zh) 2022-12-16
AU2016376034B2 (en) 2020-12-03
WO2017109679A1 (en) 2017-06-29
FI3394259T3 (fi) 2024-05-24
TW201733597A (zh) 2017-10-01
CL2019003420A1 (es) 2020-05-15
JP2021118732A (ja) 2021-08-12
AU2020267282A1 (en) 2020-12-10
CR20200609A (es) 2021-02-17
CL2018001652A1 (es) 2018-08-10
ECSP18050575A (es) 2018-07-31
AU2020267282B2 (en) 2023-05-18
CN116218848A (zh) 2023-06-06
BR112018012304A2 (pt) 2019-02-05
JP2019500428A (ja) 2019-01-10
HK1254822A1 (zh) 2019-07-26
JP2022065096A (ja) 2022-04-26
IL296844B2 (en) 2024-04-01
EA202091865A1 (ru) 2021-01-29
DOP2018000151A (es) 2018-09-15
JOP20200228A1 (ar) 2017-06-16
US20220411796A1 (en) 2022-12-29
IL296844B1 (en) 2023-12-01
CN109072237A (zh) 2018-12-21
CO2018006297A2 (es) 2018-07-10
RU2021112980A (ru) 2021-05-28
SG10201913434PA (en) 2020-03-30
JO3790B1 (ar) 2021-01-31
SV2018005714A (es) 2018-09-10
JP7027328B2 (ja) 2022-03-01
PH12018501207A1 (en) 2019-02-11
CU20180065A7 (es) 2018-11-06
CU24574B1 (es) 2022-01-13
RU2021112980A3 (ru) 2021-12-28
MY191737A (en) 2022-07-13
IL296844A (en) 2022-11-01
IL260124A (en) 2018-07-31
KR20180089517A (ko) 2018-08-08
US11473083B2 (en) 2022-10-18
RU2747734C2 (ru) 2021-05-13
PE20181448A1 (es) 2018-09-12
MX2018007726A (es) 2018-08-15
RU2018126545A (ru) 2020-01-23
AU2016376034A1 (en) 2018-07-05

Similar Documents

Publication Publication Date Title
TWI787163B (zh) 用於減少tau表現之組合物及方法
JP7354342B2 (ja) タウ発現を調節するための組成物
US11781135B2 (en) Methods for treating Alzheimer's disease
JP2017079786A (ja) α−シヌクレイン発現の調節
EP3362564B1 (en) Nucleic acid based tia-1 inhibitors
JP2018531046A6 (ja) 核酸ベースのｔｉａ−１阻害剤